Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination

Despite the promising results of new CFTR targeting drugs designed for the recovery of F508del- and class III variants activity, none of them have been approved for individuals with selected rare mutations, because uncharacterized CFTR variants lack information associated with the ability of these c...

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Detalles Bibliográficos
Autores principales: Kleinfelder, Karina, Villella, Valeria Rachela, Hristodor, Anca Manuela, Laudanna, Carlo, Castaldo, Giuseppe, Amato, Felice, Melotti, Paola, Sorio, Claudio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10299407/
https://www.ncbi.nlm.nih.gov/pubmed/37373505
http://dx.doi.org/10.3390/ijms241210358

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